A copolymer nano micelle system co-delivers genes and drugs to liver cells using galactose targeting ligands.
A functionalized nanomotor converts ultrasound energy into mechanical motion to propel through biological media and penetrate living cells.
iRNA compositions direct the RNA-induced silencing complex to degrade C5 mRNA, resolving inadequate reduction of pathological effects in disease models.
Segmented cond-sirna strands suppress background activity while maintaining high potency through preliminary duplex assembly.
Segmented delivery of guide RNAs and donor DNA templates restores CFTR function by correcting 99% of non-responsive alleles.
Engineered extracellular vesicles loaded with specific microRNA cargo resolve potency variability to deliver consistent therapeutic efficacy.
Retinoid liposomes transport siRNA to bone marrow fibroblasts, suppressing HSP47 gene expression and extracellular matrix production to treat myelofibrosis.
Magnetic core-shell nanoparticles guide CRISPR-Cas9 components into stem cells via magnetofection, bypassing viral vector immunogenicity and mutagenesis risks.
A shRNA vector targets STAT3 mRNA to inhibit intravitreal neovascularization.
Phosphate-based oligomeric compounds bind therapeutic agents and penetrate cellular membranes to enable intracellular transport.
Peptide-anionic substance complexes overcome endosomal retention barriers by facilitating direct cytoplasmic delivery of nucleic acids.
Internalizing nucleic acid molecules bind to cancer cell surface markers and mediate cellular uptake via endocytosis, reducing systemic side effects.
An RNA bridge activates a DNAzyme motor via microRNAs, enabling intracellular movement without external energy.
Protease-resistant virus-like particles protect nucleic acid cargo from degradation during purification, enabling high-efficiency separation of siRNA and mRNA.
Lipid bilayer nanopores detect nucleic acid subunits via electrical current changes.
Segmenting the polynucleotide with a localized blocker domain protects against exonuclease degradation, enabling controlled therapeutic cargo release.
Nucleic acid compositions bind cell surface mitochondrial HSP70 to internalize therapeutic agents into cancer cells.
SELEX optimization and chemical conjugation enhance binding specificity for MAGE-A3 peptides, addressing insufficient affinity of existing aptamers.
Mito-Esc phosphonium amphiphile forms self-assembled nanoparticles that protect siRNA from enzymatic degradation in biological fluids.
Enzymatically cleavable peptide linkers degrade in the small intestine to release siRNA, overcoming proteolytic barriers.
Pre-loading exosomes with ATP overcomes energy-dependent uptake limits, increasing cargo delivery by 300% while removing harmful nucleic acids.
Enzymatic removal of 5′-terminal phosphate groups from guide RNA reduces immune response and cytotoxicity during ribonucleoprotein delivery.
Segmented AAV delivery of CRISPR/Cas9 corrects CEP290 mutations, bypassing vector packaging limits.
Hairpin RNA constructs leverage endogenous Dicer processing to silence C9ORF72 transcripts, addressing manufacturing complexity of synthetic oligonucleotides.
Interfering nucleic acid molecules degrade Nurr1 mRNA in the striatum, resolving levodopa-induced dyskinesias while preserving motor function.
Modified sphingosine lipids form complexes with nucleic acids to resolve low delivery efficiency of conventional carriers.
Lipid-conjugated RNAi oligonucleotides resolve serum stability and activity trade-offs via chemical modifications, enabling effective extrahepatic delivery.
Polymer-linked oligonucleotide conjugates shield the active molecule from blood degradation and immune recognition while boosting intracellular uptake.
Replication-competent H-1PV-silencer parvovirus amplifies therapeutic shRNAs within cancer cells to overcome the short half-life of direct siRNA delivery.
Nanomaterial additives shield dsRNA bacterial solutions from photolytic breakdown during pest control applications.
Linear Polyethylenimine Hydrochloride enables lumenal loading of nucleic acid cargo into extracellular vesicles.
Heterocyclic phospholipids reduce cytotoxicity while maintaining stable nucleic acid delivery in blood circulation.
AAV vectors deliver siRNA to silence ANGPTL3, lowering triglycerides without frequent injections.
Polycation-based endosomal escape polymers disrupt membranes to resolve low efficacy and off-target effects in gene modification.
Anti-CD47 antibodies force cancer stem cell differentiation, preventing tumor recurrence and metastasis.
A three-dimensional spherical alpha-helical polypeptide complexates siRNA via hydrophobic interactions to enable efficient gene transfection.
Aptamer constructs deliver nucleic acids to beta cells, avoiding viral vector immunogenicity and enabling direct mass measurement.
Recombinant adeno-associated viruses deliver DNA encoding microRNAs to silence myotilin expression in muscle cells.
Composite lipid structures reduce cell toxicity and liver accumulation while extending circulation time for dsRNA cancer therapies.
Antibody-linked oligonucleotide complexes inhibit DUX4 expression in muscle cells by exploiting transferrin receptor-mediated uptake.
CMV promoter and miR-122 target site drive tumor-specific ribozyme expression, reducing normal tissue toxicity while maintaining high therapeutic efficiency.
DNA-RNA hybrid particles resist nuclease digestion and suppress gene expression without carrier toxicity.
Trifurcate pRNA motifs increase construct stability by optimizing base pairing, enabling reliable therapeutic delivery.
Chemically modified Neisseria meningitidis guide RNAs resolve rapid degradation and immunogenic responses to enable stable in vivo genome editing.
Engineered exosomes deliver miRNA to downregulate HER2 protein expression, reducing treatment toxicity and cost.
Pancreas-specific rAAV vectors deliver shRNA to inhibit thymidylate synthase, resolving limited treatment availability for pancreatic neuroendocrine tumors.
Ethanol-mediated spray delivers gene editing proteins directly into cells, eliminating plasmid integration risks and reducing off-target effects.